FDA approval of supplemental new drug application for Oxlumo in advanced primary hyperoxaluria type 1
Alnylam Pharmaceuticals, Inc. , the leading RNAi therapeutics company, announced that the FDA approved a label expansion for Oxlumo (lumasiran), an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in pediatric and adult patients
The approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase III study of Oxlumo in patients with severe renal impairment, including those on hemodialysis.
The FDA approval is based on positive six-month results from the ILLUMINATE-C Phase III study, in which Oxlumo treatment resulted in substantial reductions in POx and demonstrated an encouraging safety and tolerability profile in patients with compromised renal function, including those with kidney failure and undergoing treatment by hemodialysis. Elevated POx is directly related to the pathophysiology of oxalosis and results in systemic deposition of oxalate in extra-renal tissues, potentially leading to bone fractures, cardiomyopathy, impaired erythropoiesis, vision loss, skin ulcers and other serious manifestations.
The supplemental New Drug Application also included results from the open-label extensions of the ILLUMINATE-A and ILLUMINATE-B Phase III studies of pediatric and adult patients with PH1. The label has correspondingly been updated to highlight the maintenance of sustained reductions in UOx through Month 24 and Month 12, respectively.
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